Prospective Study of Rapamycin for the Treatment of SLE
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
Systemic lupus erythematosus (SLE) is an autoimmune disease of unknown origin. It involves
multiple organs including the joints, skin, kidneys and central nervous system. The disease
process is caused by a dysfunction of the immune system. The drugs currently used for the
treatment of SLE are only partially effective and carry significant risks for side-effects.
Rapamycin, also called sirolimus or Rapamune, has been approved by the FDA to prevent
rejection of organ transplants at daily doses of 2 mg to 8 mg. Patients that were resistant
or intolerant to conventional medication have been effectively treated with Rapamycin and
were able to decrease the amount of prednisone they needed.
The purpose of this study is to prospectively determine the therapeutic efficacy and
mechanism of action of Rapamune in patients with SLE. Healthy subjects not receiving Rapamune
will be asked to donate blood to serve as controls.
As part of the research effort to understand the reason for the variations in the effects of
treatment drugs by different individuals, a sub-study of the DNA makeup of subjects enrolled
in the trial will also be done. The purpose of the sub-study is to possibly determine whether
different responses to the drugs used to treat SLE have a correlation with the differences in
the genetic makeup of the subjects.
Phase:
Phase 2
Details
Lead Sponsor:
State University of New York - Upstate Medical University